Single dose of rituximab for refractory steroid-dependent nephrotic syndrome in children
Single dose of rituximab for refractory steroid-dependent nephrotic syndrome in children
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DOI：
10.1007/s00467-009-1191-0
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发表时间：
2009-07-01
影响因子：
3
通讯作者：
Iijima, Kazumoto
中科院分区：
文献类型：
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作者：
Kamei, Koichi;Ito, Shuichi;Iijima, Kazumoto
我们进行了一项多中心前瞻性试验，以评估单剂量利妥昔单抗（375 mg/m2体表面积）治疗难治性激素依赖性肾病综合征（SDNS）儿童的疗效、安全性和药代动力学。所有患者（n = 12）均能够在治疗后中位74天停用类固醇。与治疗前相比，治疗后每6个月的复发频率显著降低，每6个月的停用类固醇时间显著增加。9名患者（75%）的病情在治疗后中位数129天复发，7名患者因类固醇依赖而给予额外的利妥昔单抗。大多数复发与B细胞的恢复同时发生。然而，三名患者（25%）没有复发，B细胞恢复，疾病缓解超过1年。没有患者发生危及生命的不良事件。这是第一份关于单剂量利妥昔单抗治疗难治性SDNS的前瞻性研究报告。单剂量利妥昔单抗治疗难治性SDNS可能有效，但其预防复发的疗效在大多数患者中是短暂的。
We conducted a multicenter prospective trial to evaluate the efficacy, safety and pharmacokinetics of a single dose of rituximab (375 mg/m(2) body surface area) for the treatment of children with refractory steroid-dependent nephrotic syndrome (SDNS). All patients (n = 12) were able to discontinue steroids at a median of 74 days after treatment. The frequency of relapses per 6 months was significantly reduced and the steroid-free period per 6 months was significantly increased after treatment compared with those before treatment. The condition in nine of the patients (75%) relapsed at a median of 129 days after treatment, and seven patients were given additional rituximab due to steroid dependency. Most of the relapses developed simultaneously with recovery of B-cells. However, three patients (25%) did not have a relapse with B-cell recovery and the disease was kept in remission for more than 1 year. None of the patients developed life-threatening adverse events. This is the first report of a prospective study of a single dose of rituximab for refractory SDNS. Treatment with a single dose of rituximab may be effective for refractory SDNS, but its efficacy to prevent relapses was transient in most of the patients.